ivacaftor (Kalydeco)

Advice

Indication under review: treatment of cystic fibrosis (CF) in patients age 6 years and older who have a G551D mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.

Ivacaftor has demonstrated superiority over placebo measured by absolute change in forced expiratory volume in one second (FEV1) % predicted in two phase III, double-blind randomised studies.

The submitting company’s justification of the treatment’s cost in relation to its health benefits was not sufficient and in addition the company did not present a sufficiently robust economic assessment to gain acceptance by SMC.